Results for Gene Transfer Therapy

The research in the Sabatino Laboratory is focused on hemophilia, an inherited bleeding disorder. The interests of the laboratory include the study of variants of coagulation factor VIII to understand the biochemical properties of these proteins and to identify novel variants with enhanced function, and the development of gene-based therapeutic approaches for treating hemophilia.

Dr. Margaritis uses biochemical, molecular, and complex in vivo methodology within the field of coagulation to advance the understanding of molecular mechanisms involved in pro- and anti-coagulant reactions, and translate research for the treatment of coagulation defects.

Dr. Yudkoff's research is focused on the use of stable isotopes to measure the rate of production of urea in humans who have a urea cycle defect, or a congenital inability to detoxify ammonia into urea.

Dr. Breda is developing safe, potentially curative genetic therapies for sickle cell disease and b-thalassemia.